Industry Updates

Cure SMA Announces Expanded Phase 3 of Real World Evidence Collaboration

July 31, 2023
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Cure SMA is pleased to announce the launch of an expanded Phase 3 of the Real World Evidence Collaboration. The collaboration was established to leverage […]

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New Data at Cure SMA Highlight Potential Benefit of SPINRAZA® (nusinersen) in Infants and Toddlers with Unmet Clinical Needs After Gene Therapy

July 1, 2023
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Biogen Inc. announced new SPINRAZA® (nusinersen) data aimed at answering critical questions for the spinal muscular atrophy (SMA) community. The data were presented at the SMA […]

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Four-Year Follow-Up Data for Genentech’s Evrysdi Show Continued Increase in Number of Children With a Severe Form of Spinal Muscular Atrophy (SMA) Able to Sit, Stand and Walk

July 1, 2023
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Genentech, a member of the Roche Group, announced today new long-term data for Evrysdi® (risdiplam) from the open-label extension (n=50) of the pivotal FIREFISH study, reinforcing […]

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New 36-Month Apitegromab Extension Data Reinforce Long-Term Substantial and Sustained Improvement of Motor Function in Phase 2 TOPAZ Trial Patients with Nonambulatory Spinal Muscular Atrophy

June 30, 2023
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Below is an excerpt from a Scholar Rock press release: Scholar Rock, a Phase 3, clinical-stage biopharmaceutical company focused on the treatment of serious diseases […]

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Community Update from Genentech

June 22, 2023
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We are pleased to share the following SMA community letter from our partners at Genentech on their clinical development programs and initiatives. Dear SMA Community, […]

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Novartis Gene Therapies Release May 2023 SMA Community Update

June 7, 2023
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Novartis Gene Therapies recently provided an update on their clinical development program and initiatives. We invite you to check it out here!

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Biogen Releases Q1 2023 Community Statement

May 15, 2023
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Biogen recently issued a Q1 SMA Community Statement. Check it out here!

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Novartis shares Zolgensma long-term data demonstrating sustained durability up to 7.5 years post-dosing; 100% achievement of all assessed milestones in children treated prior to SMA symptom onset

April 11, 2023
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Children in LT-001 treated after SMA symptom onset maintained or achieved additional milestones up to 7.5 years post one-time intravenous infusion All children (100%) in […]

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Biohaven’s Taldefgrobep Alfa Receives FDA Fast Track Designation for Spinal Muscular Atrophy

February 24, 2023
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This week, Biohaven announced that it received Fast Track designation from the U.S. Food and Drug Administration (FDA) for taldefgrobep alfa, a novel anti-myostatin adnectin, […]

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Genentech Releases SMA Community Letter

December 30, 2022
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Our industry partners at Genentech recently released an SMA community letter summarizing 2022 developments and initiates. Check it out here!

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